Sample Size Calculations in Clinical Research, Second Edition
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Summary
Focusing on an integral part of pharmaceutical development, Sample Size Calculations in Clinical Research, Second Edition presents statistical procedures for performing sample size calculations during various phases of clinical research and development. It provides sample size formulas and procedures for testing equality, noninferiority/superiority, and equivalence.
A comprehensive and unified presentation of statistical concepts and practical applications, this book highlights the interactions between clinicians and biostatisticians, includes a well-balanced summary of current and emerging clinical issues, and explores recently developed statistical methodologies for sample size calculation. Whenever possible, each chapter provides a brief history or background, regulatory requirements, statistical designs and methods for data analysis, real-world examples, future research developments, and related references.
One of the few books to systematically summarize clinical research procedures, this edition contains new chapters that focus on three key areas of this field. Incorporating the material of this book in your work will help ensure the validity and, ultimately, the success of your clinical studies.
Table of Contents
Preface
Introduction
Regulatory Requirement
Basic Considerations
Procedures for Sample Size Calculation
Aims and Structure of the Book
Considerations Prior to Sample Size Calculation
Confounding and Interaction
One-Sided Test versus Two-Sided Test
Crossover Design versus Parallel Design
Subgroup/Interim Analyses
Data Transformation
Practical Issues
Comparing Means
One-Sample Design
Two-Sample Parallel Design
Two-Sample Crossover Design
Multiple-Sample One-Way ANOVA
Multiple-Sample Williams Design
Practical Issues
Large Sample Tests for Proportions
One-Sample Design
Two-Sample Parallel Design
Two-Sample Crossover Design
One-Way Analysis of Variance
Williams Design
Relative Risk—Parallel Design
Relative Risk—Crossover Design
Practical Issues
Exact Tests for Proportions
Binomial Test
Fisher’s Exact Test
Optimal Multiple-Stage Designs for Single Arm Trials
Flexible Designs for Multiple-Arm Trials
Remarks
Tests for Goodness-of-Fit and Contingency Tables
Tests for Goodness-of-Fit
Test for Independence—Single Stratum
Test for Independence—Multiple Strata
Test for Categorical Shift
Carry-Over Effect Test
Practical Issues
Comparing Time-to-Event Data
Basic Concepts
Exponential Model
Cox’s Proportional Hazards Model
Weighted Log-Rank Test
Practical Issues
Group Sequential Methods
Pocock’s Test
O’Brien and Fleming’s Test
Wang and Tsiatis’ Test
Inner Wedge Test
Binary Variables
Time-to-Event Data
Alpha Spending Function
Sample Size Re-Estimation
Conditional Power
Practical Issues
Comparing Variabilities
Comparing Intra-Subject Variabilities
Comparing Intra-Subject CVs
Comparing Inter-Subject Variabilities
Comparing Total Variabilities
Practical Issues
Bioequivalence Testing
Bioequivalence Criteria
Average Bioequivalence
Population Bioequivalence
Individual Bioequivalence
In Vitro Bioequivalence
Dose Response Studies
Continuous Response
Binary Response
Time-to-Event Endpoint
Williams’ Test for Minimum Effective Dose (MED)
Cochran–Armitage’s Test for Trend
Dose Escalation Trials
Concluding Remarks
Microarray Studies
Literature Review
False Discovery Rate (FRD) Control
Family-Wise Error Rate (FWER) Control
Concluding Remarks
Bayesian Sample Size Calculation
Posterior Credible Interval Approach
Posterior Error Approach
The Bootstrap-Median Approach
Concluding Remarks
Nonparametrics
Violation of Assumptions
One-Sample Location Problem
Two-Sample Location Problem
Test for Independence
Practical Issues
Sample Size Calculation in Other Areas
QT/QTc Studies with Time-Dependent Replicates
Propensity Analysis in Nonrandomized Studies
ANOVA with Repeated Measures
Quality of Life
Bridging Studies
Vaccine Clinical Trials
INDEX
Editorial Reviews
"… an excellent resource, and the added material (which amounts to roughly 100 pages) more than justifies the publication of the second edition."
—Joseph Cavanaugh, University of Iowa, Journal of the America Statistical Association, March 2009
"The second edition book presents on a total of 465 pages a comprehensive derivation of sample size formulae for a wide variety of testing scenarios from many different clinical research topics … well written and easy to read due to the uniform organizational structure of all chapters. Each chapter contains a detailed description of the statistical test problems, the adequate statistical formulae for sample size calculations, numerical examples as well as practical issues … this book is recommended to biostatisticians and clinical scientists in medical or pharmaceutical research and provides a comprehensive reference of sample size calculations covering a wide range of different clinical research topics."
—Iris Burkholder, Zentralblatt MATH, 2009
"…The second edition has been carefully updated from the first edition. … One of the strengths of the book is the organizational structure. Each chapter contains comprehensive references, examples, and practical considerations. The book is clearly laid out and easy to read. The table of contents and the index are comprehensive, which makes the book quite useful as a reference. … In summary, this is a useful, comprehensive compendium of almost every possible sample size formula. The strong organization and carefully defined formulae will aid any researcher designing a study."
—Biometrics, December 2008
“This impressive book contains formulae for computing sample size in a wide range of settings. One-sample studies and two-sample comparisons for quantitative, binary, and time-to-event outcomes are covered comprehensively, with separate sample size formulae for testing equality, non-inferiority, and equivalence. Many less familiar topics are also covered, including sample size for comparing k samples, bioequivalence, dose–response studies, and (new in this second edition) microarray studies and Bayesian sample size determination. … Overall, this is a useful reference for the mathematical statistician …”
—Ian White (Medical Research Council Biostatistics Unit), Journal of the Royal Statistical Society
"… The book is well written and easy to read. … a useful comprehensive reference of sample size calculation procedures for clinical research. It should be a valuable reference book for biostatisticians and clinical scientists in medical or pharmaceutical research."
—Statistics in Medicine, 2008
"This book gives excellent coverage to the many types of study design and aims that are likely to be encountered. The main strength of the book is the vast collection of sample size calculations from many different areas of clinical research … an excellent reference for people involved in the designing of clinical research projects."
—The Journal of Perioperative Practice, September 2008
"…This text provides procedures and formulae for the determination of sample size and the appropriate calculation of power for the hypotheses that reflect study objectives under a valid study design. … this second edition will appeal to both practitioners and students alike."
—International Statistical Review, 2008
Praise for the First Edition
“This well-composed book … contains sample size formulas and examples. … a good reference book for researchers in clinical trials.”
—Journal of Statistical Computation and Simulation, Vol. 74, No. 5, May 2004
“The reference list contains details of an excellent collection of articles. The examples are clearly illustrated. This is a fascinating book, and applied statisticians and health and medical researchers will like it a lot. Statistical consultants will be fond of the book as a reference guide.”
—Journal of Statistical Computation and Simulation, Vol. 75, No. 9, September 2005
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